Clinical expert panel on monitoring potential lung toxicity of inhaled oligonucleotides: Consensus points and recommendations

Nucleic Acid Therapeutics

Volumn 22, Issue 4, 2012, Pages 246-254

  Alton, Eric W ^a   Boushey, Homer A ^b   Garn, Holger ^c   Green, Francis H ^d   Hodges, Michael ^e   Martin, Richard J ^f   Murdoch, Robert D ^g   Renz, Harald ^h   Shrewsbury, Stephen B ⁱ   Seguin, Rosanne ^j   Johnson, Graham ⁱ   Parry, Joel D ^g   Tepper, Jeff ^k   Renzi, Paolo ^l   Cavagnaro, Joy ^m   Ferrari, Nicolay ^j  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c Sterna Biologicals   (Germany)

^d UNIVERSITY OF CALGARY   (Canada)

^e Altair Therapeutics   (United States)

^f DEPARTMENT OF MEDICINE   (United States)

^g GLAXOSMITHKLINE   (United Kingdom)

^h PHILIPPS UNIVERSITY MARBURG   (Germany)

ⁱ AVI BIOPHARMA INC   (United States)

^j INSTITUTE FOR RESEARCH IN IMMUNOLOGY AND CANCER   (Canada)

^k Tepper Nonclinical Consulting   (United States)

^l UNIVERSITÉ DE MONTRÉAL   (Canada)

^m Access BIO   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

8 ISOPROSTANE; C REACTIVE PROTEIN; CARBON MONOXIDE; HYDROCARBON; INTERLEUKIN 6; INTERLEUKIN 8; MONOCYTE CHEMOTACTIC PROTEIN 1; NITRIC OXIDE; OLIGONUCLEOTIDE;

ARTICLE; ARTIFICIAL VENTILATION; BLOOD GAS; CAPILLARY PERMEABILITY; CHRONIC OBSTRUCTIVE LUNG DISEASE; COMPUTER ASSISTED TOMOGRAPHY; CYSTIC FIBROSIS; EXHALATION; FORCED EXPIRATORY FLOW; FORCED EXPIRATORY VOLUME; FORCED VITAL CAPACITY; HISTOLOGY; HUMAN; LUNG CAPACITY; LUNG FUNCTION; LUNG LAVAGE; LUNG TOXICITY; MACROPHAGE ACTIVATION; NONHUMAN; PEAK EXPIRATORY FLOW; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; RESPIRATORY TRACT DISEASE; SPIROMETRY; SPUTUM ANALYSIS; TRANSBRONCHIAL BIOPSY; TREATMENT RESPONSE;

ADMINISTRATION, INHALATION; ANIMALS; BIOLOGICAL MARKERS; DRUG EVALUATION, PRECLINICAL; HUMANS; LUNG; OLIGONUCLEOTIDES; PRACTICE GUIDELINES AS TOPIC; RESPIRATORY FUNCTION TESTS;

EID: 84865412347     PISSN: 21593337     EISSN: None     Source Type: Journal    
DOI: 10.1089/nat.2012.0345     Document Type: Article

References (48)

1. ALI, S., LEONARD, S.A., KUKOLY, C.A., JAMES METZGER, W., WOOLES, W.R., MCGINTY, J.F., TANAKA, M., SANDRASAGRA, A., and NYCE, J.W. (2001). Absorption, distribution, metabolism, and excretion of a respirable antisense oligonucleotide for asthma. Am. J. Respir. Crit. Care Med. 163, 989-993. (Pubitemid 32275876)
2. ATTILI, A.K., KAZEROONI, E.A., GROSS, B.H., FLAHERTY, K.R., MYERS, J.L., and MARTINEZ, F.J. (2008). Smoking-related interstitial lung disease: radiologic-clinical-pathologic correlation1. Radiographics 28, 1383-1396.
3. BALL, H., SANDRASAGRA, A., TANG, L., VAN SCOTT, M., WILD, J., and NYCE, J. (2003). Clinical potential of respirable antisense oligonucleotides (RASONs) in asthma. Am. J. Pharmacogenomics 3, 97-106. (Pubitemid 36628604)
4. BELDA, J., LEIGH, R., PARAMESWARAN, K., O'BYRNE, P.M., SEARS, M.R., and HARGREAVE, F.E. (2000). Induced sputum cell counts in healthy adults. Am. J. Respir. Crit. Care Med. 161, 475-478. (Pubitemid 30129455)
5. BERRY, M., MORGAN, A., SHAW, D.E., PARKER, D., GREEN, R., BRIGHTLING, C., BRADDING, P., WARDLAW, A.J., and PAVORD, I.D. (2007). Pathological features and inhaled corticosteroid response of eosinophilic and non-eosinophilic asthma. Thorax 62, 1043-1049. (Pubitemid 350206195)
6. BLANC, P.D., WONG, H., BERNSTEIN, M.S., and BOUSHEY, H.A. (1991). An experimental human model of metal fume fever. Ann. Intern. Med. 114, 930-936.
7. BRAIN, J.D. (2007). Inhalation, deposition, and fate of insulin and other therapeutic proteins, Diabetes Technol. Ther. 9, S4-S15.
8. BRASEY, A., IGUE, R., DJEMAME, L., SEGUIN, S., RENZI, P.M., FERRARI, N., and SEGUIN, R. (2011). The effect of in vitro exposure to antisense oligonucleotides on macrophage morphology and function. J. Nucleic Acids Investig. 2, e12.
9. COSTA, D.L., KUTZMAN, R.S., LEHMANN, J.R., and DREW, R.T. (1986). Altered lung function and structure in the rat after subchronic exposure to acrolein. Am. Rev. Respir. Dis. 133, 286-291. (Pubitemid 16123925)
10. CROOKE, S.T. (1998). Vitravene: another piece in the mosaic.Antisense Nucleic Acid Drug Dev. 8, 7-8.
11. DEVINCENZO, J., LAMBKIN-WILLIAMS, R., WILKINSON, T., CEHELSKY, J., NOCHUR, S., WALSH, E., MEYERS, R., GOLLOB, J., and VAISHNAW, A. (2010). A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus. Proc. Natl. Acad. Sci. U. S. A. 107, 8800-8805.
12. DEVINCENZO, J., CEHELSKY, J.E., ALVAREZ, R., ELBASHIR, S., HARBORTH, J., TOUDJARSKA, I., NECHEV, L., MURUGAIAH, V., VLIET, A.V., VAISHNAW, A.K., and MEYERS, R. (2008). Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV). Antiviral Res. 77, 225-231.
13. DIA/FDA/HEALTH CANADA/AAPS/OTS. (2010). 3rd Oligonucleotide based therapeutics conference-where regulators and industry partner to advance oligonucleotide science together. March 23-25, 2010, Bethesda MD.
14. DOE, C., BAFADHEL, M., SIDDIQUI, S., DESAI, D., MISTRY, V., RUGMAN, P., MCCORMICK, M., WOODS, J., MAY, R., et al. (2010). Expression of the T helper 17-associated cytokines IL-17A and IL-17F in asthma and COPD. Chest 138, 1140-1147.
15. DREWEK, R., GARBER, E., STANCLIK, S., SIMPSON, P., NUGENT, M., and GERSHAN, W. (2009). The FEF25-75 and its decline as a predictor of methacholine responsiveness in children. J. Asthma 46, 375-381.
16. GAUVREAU, G.M., HESSEL, E.M., BOULET, L.-P., COFFMAN, R.L., and O'BYRNE, P.M. (2006). Immunostimulatory sequences regulate interferon-inducible genes but not allergic airway responses. Am. J. Respir. Crit. Care Med. 174, 15-20. (Pubitemid 43955239)
17. GAUVREAU, G.M., BOULET, L.P., COCKCROFT, D.W., BAATJES, A., COTE, J., DESCHESNES, F., DAVIS, B., STRINICH, T., HOWIE, K., et al. (2008). Antisense therapy against CCR3 and the common beta chain attenuates allergen-induced eosinophilic responses. Am. J. Respir. Crit. Care Med. 177, 952-958. (Pubitemid 351717588)
18. GREEN, J.D. (1994). Pharmaco-toxicological expert report pulmozyme rhDNase Genentech, Inc. Hum. Exp. Toxicol. 13, S1-S42.
19. GUIMOND, A., VIAU, E., AUBE, P., RENZI, P.M., PAQUET, L., and FERRARI, N. (2008). Advantageous toxicity profile of inhaled antisense oligonucleotides following chronic dosing in non-human primates. Pulm. Pharmacol. Ther. 21, 845-854.
20. HILLAS, G., LOUKIDES, S., KOSTIKAS, K., and BAKAKOS, P. (2009). Biomarkers obtained by non-invasive methods in patients with COPD: where do we stand, what do we expect? Curr. Med. Chem. 16, 2824-2838.
21. HSIA, C.C. (2002). Recruitment of lung diffusing capacity. Chest 122, 1774-1783.
22. KELLY, M.M., LEIGH, R., and HARGREAVE, F. (2000a). Validation of assays for inflammatory mediators in sputum. Eur. Respir. J. 16, 1208-1209. (Pubitemid 32148140)
23. KELLY, M.M., HARGREAVE, F.E., and COX, G. (2003). A method to preserve sputum for delayed examination. Eur. Respir. J. 22, 996-1000. (Pubitemid 37540655)
24. KELLY, M.M., LEIGH, R., MCKENZIE, R., KAMADA, D., RAMSDALE, E.H., and HARGREAVE, F.E. (2000b). Induced sputum examination: diagnosis of pulmonary involvement in Fabry's disease. Thorax 55, 720-721. (Pubitemid 30468232)
25. KELLY, M.M., LEIGH, R., JAYARAM, L., GOLDSMITH, C.H., PARAMESWARAN, K., and HARGREAVE, F.E. (2006). Eosinophilic bronchitis in asthma: a model for establishing doseresponse and relative potency of inhaled corticosteroids. J. Allergy Clin. Immunol. 117, 989-994.
26. KELLY, M.M., KEATINGS, V., LEIGH, R., PETERSON, C., SHUTE, J., VENGE, P., and DJUKANOVIC , R. (2002). Analysis of fluid phase mediators. Eur. Respir. J. 37, 24s-39s.
27. KELLY, M.M., O'CONNOR, T.M., LEIGH, R., OTIS, J., GWOZD, C., GAUVREAU, G.M., GAULDIE, J., and O'BYRNE, P.M. (2010). Effects of budesonide and formoterol on allergeninduced airway responses, inflammation, and airway remodeling in asthma. J. Allergy Clin. Immunol. 125, 349-356.e13.
28. KING, G.G. (2011). Cutting edge technologies in respiratory research: lung function testing. Respirology 16, 883-890.
29. KRIEG, A. (2000). The role of CpG motifs in innate immunity. Curr. Opin. Immunol. 12, 35-43. (Pubitemid 30093448)
30. LEHTIMAKI, L., KANKAANRANTA, H., SAARELAINEN, S., HAHTOLA, P., JARVENPAA, R., KOIVULA, T., TURJANMAA, V., and MOILANEN, E. (2001). Extended exhaled NO measurement differentiates between alveolar and bronchial inflammation. Am. J. Respir. Crit. Care Med. 163, 1557-1561. (Pubitemid 32578755)
31. LEVIN, A.A. (1999). A review of the issues in the pharmacokinetics and toxicology of phosphorothioate antisense oligonucleotides. Biochim. Biophys. Acta 1489, 69-84.
32. LEVIN, A.A., HENRY, S.P., MONTEITH, D.K., and TEMPLIN, M. (2001). Toxicity of antisense oligonucleotides. In: Antisense Drug Technology: Principles, Strategies, and Applications. S.T. Crooke, ed. (Marcel Dekker, Inc., New York), pp. 201-267.
33. LEVIN, A.A., MONTEITH, D.K., LEEDS, J.M., NICKLIN, P.L., GEARY, R.S., BUTLER, M., TEMPLIN, M.V., and HENRY, S.P. (1998). Toxicity of oligonucleotide therapeutic agents. In: Handbook of Experimental Pharmacology. G.V.R Born et. al., eds. (Springer-Verlag, New York), pp. 169-215.
34. LEWIS, R.W., BILLINGTON, R., DEBRYUNE, E., GAMER, A., LANG, B., and CARPANINI, F. (2002). Recognition of adverse and nonadverse effects in toxicity studies. Toxicol. Pathol. 30, 66-74. (Pubitemid 34194560)
35. LIU, M.C., RIESE, R.J., VAN GUNDY, K., NORWOOD, P., SULLIVAN, B.E., SCHWARTZ, P.F., and TEETER, J.G. (2008). Effects of inhaled human insulin on airway lining fluid composition in adults with diabetes. Eur. Resp. J. 32, 180-188.
36. MARTINEZ, F.J., and FLAHERTY, K. (2006). Pulmonary function testing in idiopathic interstitial pneumonias. Proc. Am. Thorac. Soc. 3, 315-321. (Pubitemid 43845408)
37. MOSSER, D.M., and EDWARDS, J.P. (2008). Exploring the full spectrum of macrophage activation. Nat. Rev. Immunol. 8, 958-969.
38. NAGATANI, Y., NITTA, N., OTANI, H., MUKAISHO, K., SONODA, A., NITTA-SEKO, A., TAKAHASHI, M., and MURATA, K. (2011). Quantitative measurement of bleomycininduced lung fibrosis in rabbits using sequential in vivo regional analysis and high-resolution computed tomography: correlation with pathologic findings. Acad. Radiol. 18, 672-681.
39. NG, E.W., and ADAMIS, A.P. (2005). Targeting angiogenesis, the underlying disorder in neovascular age-related macular degeneration. Can. J. Ophthalmol. 40, 352-368. (Pubitemid 40942262)
40. SAHA, S., DOE, C., MISTRY, V., SIDDIQUI, S., PARKER, D., SLEEMAN, M., COHEN, E. S., and BRIGHTLING, C.E. (2009). Granulocyte-macrophage colony-stimulating factor expression in induced sputum and bronchial mucosa in asthma and COPD. Thorax 64, 671-676.
41. SAZANI, P., WELLER, D.L., and SHREWSBURY, S.B. (2010). Safety pharmacology and genotoxicity evaluation of AVI- 4658. Int. J. Toxicol. 29, 143-156.
42. TASK FORCE, ESOP. (1990). Clinical guidelines and indications for bronchoalveolar lavage (BAL): report of the European Society of Pneumology task group on BAL. Eur. Respir. J. 3, 937-976.
43. TEMPLIN, M., LEVIN, A., GRAHAM, M., ABERG, P., AXELSSON, B., BUTLER, M., GEARY, R., and BENNETT, C. (2000). Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice. Antisense Nucleic Acid Drug Dev. 10, 359-368.
44. in 't VEEN, J.C., BEEKMAN, A.J., BEL, E. H., and Sterk, P.J. (2000). Recurrent exacerbations in severe asthma are associated with enhanced airway closure during stable episodes. Am. J. Respir. Crit. Care Med. 161, 1902-1906. (Pubitemid 30418127)
45. VERHOEVEN, G.T., WIJKHUIJS, A.J., HOOIJKAAS, H., HOOGSTEDEN, H.C., and SLUITER, W. (2000). Effect of an inhaled glucocorticoid on reactive oxygen species production by bronchoalveolar lavage cells from smoking COPD patients. Mediators Inflamm. 9, 109-113. (Pubitemid 30482435)
46. VERSCHAKELEN, J.A. (2010). The role of high-resolution computed tomography in the work-up of interstitial lung disease. Curr. Opin. Pulm. Med. 16, 503-510. DOI:10.1097/ MCP.0b013e32833cc997.
47. VLACHOS-MAYER, H., LEIGH, R., SHARON, R., HUSSACK, P., and HARGREAVE, F. (2000). Success and safety of sputum induction in the clinical setting. Eur. Resp. J. 16, 997-1000. (Pubitemid 32049393)
48. ZHANG, J., YAO, X., YU, R., BAI, J., SUN, Y., HUANG, M., ADCOCK, I., and BARNES, P. (2010). Exhaled carbon monoxide in asthmatics: a meta-analysis. Resp. Res. 11, 50.